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Gene therapy using haematopoietic stem and progenitor cells

Ferrari, G; Thrasher, AJ; Aiuti, A; (2021) Gene therapy using haematopoietic stem and progenitor cells. Nature Reviews Genetics , 22 (4) pp. 216-234. 10.1038/s41576-020-00298-5. Green open access

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Abstract

Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective treatment modality for monogenic disorders of the blood system such as primary immunodeficiencies and β-thalassaemia. Medicinal products based on autologous HSPCs corrected using lentiviral and gammaretroviral vectors have now been approved for clinical use, and the site-specific genome modification of HSPCs using gene editing techniques such as CRISPR–Cas9 has shown great clinical promise. Preclinical studies have shown engineered HSPCs could also be used to cross-correct non-haematopoietic cells in neurodegenerative metabolic diseases. Here, we review the most recent advances in HSPC gene therapy and discuss emerging strategies for using HSPC gene therapy for a range of diseases.

Type: Article
Title: Gene therapy using haematopoietic stem and progenitor cells
Open access status: An open access version is available from UCL Discovery
DOI: 10.1038/s41576-020-00298-5
Publisher version: https://doi.org/10.1038/s41576-020-00298-5
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10128384
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